Of Pfizer, a New Treatment for a Rare Disease and Carrot Cells
- Wednesday, December 2, 2009, 0:00
- Health News
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Pfizer’s deal today with Israeli biotech Protalix BioTherapeutics to license most of the rights for a promising therapy for a rare disease caught our eye for a number of reasons:
The deal gives Pfizer rights to sell a treatment for Gaucher’s disease that will soon go for FDA approval. Pfizer will pay Protalix as much as $115 million for rights to sell the therapy anywhere but Israel. The dollar amount is minuscule for a big pharma with a $151 billion market cap, but Pfizer has decided to break the company into smaller units functioning relatively independently. The agreement represents the first move by Pfizer’s Established Products Business unit into rare and orphan diseases, a space traditionally inhabited by biotechs. David Simmons, who heads the unit, said Pfizer had decided in the last year to pursue opportunities in rare diseases like Gaucher’s. “It’s not a blockbuster strategy,” he said.
It is a strategy that puts Pfizer in direct competition with Genzyme, which the WSJ reported has made a living out of selling products that are temporarily shielded from drug wars because they treat rare diseases. By definition, there aren’t many patients for rare diseases, but drug makers can charge a premium for remedies. Genzyme’s treatment for Gaucher disease, called Cerezyme, can cost patients as much as $300,000 a year. Cerezyme had $1.2 billion in sales in 2008. But production problems have depressed sales this year, opening the way for rivals. “What we want to do is offer an alternative,” said Protalix CEO David Aviezer. (Genzyme, by the way, said today that it had resumed shipping Cerezyme vials made in the plant that had contamination problems.)
The Protalix treatment is one of those biologics that is grown through a complicated process in living cells, in this case carrot cells. The cells are genetically engineered to produce the therapy. They do so in vats, or bioreactors. “It’s kind of like, if you will, carrot juice growing in these bioreactors,” Aviezer said. He added that the therapy, whose scientific moniker is taliglucerase alfa, would be the first on the market to be made in plant cells. Aviezer said Protalix has been working on the technology for more than a decade and hopes to use it to make remedies for other rare diseases. The deal doesn’t give Pfizer rights to the technology, but Pfizer’s Simmons made clear during an interview that the drug maker thinks highly of it and is interested in future applications.
Photo: Associated Press
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